PL3411484T3 - Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego - Google Patents

Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego

Info

Publication number
PL3411484T3
PL3411484T3 PL17747935.9T PL17747935T PL3411484T3 PL 3411484 T3 PL3411484 T3 PL 3411484T3 PL 17747935 T PL17747935 T PL 17747935T PL 3411484 T3 PL3411484 T3 PL 3411484T3
Authority
PL
Poland
Prior art keywords
adenosatellite
virus
complementary
threaded
injection
Prior art date
Application number
PL17747935.9T
Other languages
English (en)
Inventor
Anthony DONSANTE
Karen Kozarsky
Nicholas Matthew Boulis
Jonathan Patrick Riley
Original Assignee
Emory University
Regenxbio Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Emory University, Regenxbio Inc. filed Critical Emory University
Publication of PL3411484T3 publication Critical patent/PL3411484T3/pl

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL17747935.9T 2016-02-05 2017-01-25 Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego PL3411484T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662292157P 2016-02-05 2016-02-05
PCT/US2017/014914 WO2017136202A1 (en) 2016-02-05 2017-01-25 Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid

Publications (1)

Publication Number Publication Date
PL3411484T3 true PL3411484T3 (pl) 2024-02-19

Family

ID=59500060

Family Applications (1)

Application Number Title Priority Date Filing Date
PL17747935.9T PL3411484T3 (pl) 2016-02-05 2017-01-25 Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego

Country Status (15)

Country Link
US (3) US12070510B2 (pl)
EP (2) EP3411484B1 (pl)
JP (4) JP2019504888A (pl)
AU (1) AU2017214283B2 (pl)
DK (1) DK3411484T3 (pl)
ES (1) ES2962860T3 (pl)
FI (1) FI3411484T3 (pl)
HR (1) HRP20231451T1 (pl)
HU (1) HUE064408T2 (pl)
IL (2) IL300254B2 (pl)
LT (1) LT3411484T (pl)
PL (1) PL3411484T3 (pl)
PT (1) PT3411484T (pl)
SI (1) SI3411484T1 (pl)
WO (1) WO2017136202A1 (pl)

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CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
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Also Published As

Publication number Publication date
DK3411484T3 (da) 2023-11-20
JP2025041933A (ja) 2025-03-26
IL300254A (en) 2023-03-01
US12070510B2 (en) 2024-08-27
AU2017214283A1 (en) 2018-08-02
EP3411484A4 (en) 2019-08-07
PT3411484T (pt) 2023-11-17
AU2017214283B2 (en) 2023-06-29
SI3411484T1 (sl) 2023-12-29
JP2019504888A (ja) 2019-02-21
IL300254B2 (en) 2025-06-01
EP3411484B1 (en) 2023-10-04
EP4299079A2 (en) 2024-01-03
WO2017136202A1 (en) 2017-08-10
FI3411484T3 (fi) 2023-11-15
EP4299079A3 (en) 2024-05-01
HRP20231451T1 (hr) 2024-03-01
IL300254B1 (en) 2025-02-01
ES2962860T3 (es) 2024-03-21
HUE064408T2 (hu) 2024-03-28
CA3012653A1 (en) 2017-08-10
IL260954B2 (en) 2023-07-01
JP2022101648A (ja) 2022-07-06
IL260954A (pl) 2018-09-20
IL260954B1 (en) 2023-03-01
LT3411484T (lt) 2023-11-27
EP3411484A1 (en) 2018-12-12
US20190038777A1 (en) 2019-02-07
US20240108761A1 (en) 2024-04-04
JP2024084797A (ja) 2024-06-25
US20250135041A1 (en) 2025-05-01

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